Universität zu Köln

Al Kabbani, Mohamed Aghyad, Wunderlich, Gilbert, Koeler, Christoph and Zempel, Hans (2022). AAV-based gene therapy approaches for genetic forms of tauopathies and related neurogenetic disorders. Biocell, 46 (4). S. 847 - 854. HENDERSON: TECH SCIENCE PRESS. ISSN 1667-5746

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Abstract

Tauopathies comprise a spectrum of genetic and sporadic neurodegenerative diseases mainly characterized by the presence of hyperphosphorylated TAU protein aggregations in neurons or glia. Gene therapy, in particular adeno-associated virus (AAV)-based, is an effective medical approach for difficult-to-treat genetic diseases for which there are no convincing traditional therapies, such as tauopathies. Employing AAV-based gene therapy to treat, in particular, genetic tauopathies has many potential therapeutic benefits, but also drawbacks which need to be addressed in order to successfully and efficiently adapt this still unconventional therapy for the various types of tauopathies. In this Viewpoint, we briefly introduce some potentially treatable tauopathies, classify them according to their etiology, and discuss the potential advantages and possible problems of AAV-based gene therapy. Finally, we outline a future vision for the application of this promising therapeutic approach for genetic and sporadic tauopathies.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Al Kabbani, Mohamed Aghyad UNSPECIFIED UNSPECIFIED UNSPECIFIED Wunderlich, Gilbert UNSPECIFIED UNSPECIFIED UNSPECIFIED Koeler, Christoph UNSPECIFIED UNSPECIFIED UNSPECIFIED Zempel, Hans UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-656872
DOI:	10.32604/biocell.2022.018144
Journal or Publication Title:	Biocell
Volume:	46
Number:	4
Page Range:	S. 847 - 854
Date:	2022
Publisher:	TECH SCIENCE PRESS
Place of Publication:	HENDERSON
ISSN:	1667-5746
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language ADENOASSOCIATED VIRUS; EFFICIENT TRANSDUCTION; VIRAL VECTORS; MOUSE MODEL; DELIVERY; TAU; EXPRESSION; TOXICITY; PRIMATES Multiple languages Biology Multiple languages
URI:	http://kups.ub.uni-koeln.de/id/eprint/65687